In the past few years,China has pushed forward the availability of diagnosis and treatment of rare diseases.However,only 5％of patients receive effective treatment in the world.In China,the difficul-ties in having access to drugs against rare disease is still one of the major problems in medical service and the main cause of patients complaints for"difficulties in getting treatment"and"difficulties in getting drugs".The availability of the medication for rare disease is a coordinating mechanism involving multiple such points in the chain as in research and development,production,circulation,usage,way of payment,technology in-novation and so forth.It also relates to the actors in supply,demand,and policy decision-makers.The com-plexity is different from that of common drugs.This article provides a brief overview of the current status of availability policies of medication for rare diseases in China and in other countries,as well as the technology in drug development.In addition,the article points out the problems,challenges and expectations for future possibilities.
关键词
罕见病/孤儿药/用药保障/药品支付/审评审批
Key words
rare diseases/orphan drugs/guarantee for drugs/drug paymemnt/audit and approve
维普
万方数据